Candlelighters Childhood Cancer Foundation

1
Candlelighters Childhood Cancer Foundation

• FDA
• ODAC Subcommittee
• October 17, 2002

2
Patient/Family Perspective

• Not derived from a formal survey
• Prospective survey possible
• Shared perspective
• Personal experience
• parent of child with AML (1987)
• professional interactions with thousands of
  families.

3
Constituency

• Candlelighters National Office
• 32 years of serving families information
  resource, referrals, and support.
• 6,000 phone calls/year
• 14,000 email queries/year
• 155,000 web site visitors/year
• (1.5 million hits).

4
Specific Inquiries

• Approximately HALF queries
• Clinical trial information, availability
• Institutional referrals
• Referral to disease specialists

5
HopeLink

• HopeLink clinical trial search engine on website
  (www.candlelighters.org)
• 385 childhood cancer trials listed, currently
  open for enrollment.
• Comprehensive trial listings standard and
  medical formats
• eligibility criteria
• investigative site locations
• contact information

6
Hope

• When the doctor explained to us about Melissa's
  leukemia, he said that APML is incurable and its
  very rare and very deadly. Can you give us hope?

7
What do families want?

• Magic bullet to cure child with resistant
  disease
• Options for therapies
• Neuroblastoma IV
• Brain tumors
• Metastatic solid tumors
• AML

8

• The childhood cancer population is a small
  community in number, but large in spirit and used
  to success. The clinical trial process is what
  has brought pediatric oncology the cures that
  give hope and help to parents and survivors, and
  has created a foundation of trust upon which to
  build improved and novel treatments.
• Grace Monaco, Founder CCCF

9
Timing Access to New Drugs

• Use of new drugs in recurrent/refractory cases
• Accurate information built on foundation of
  trust
• Relative safety.
• Pre-clinical models, animal testing, adult
  testing.
• Probable tumor response
• Possible magic bullet/cure vs. actual small
  percentage response rate to Phase I trials.
• Side effects of treatment
• Impact on quality of life at probable end of
  life.

10
Balance in Decision Making

• Feedback from families in phase 1 trial
  experiences (end of life)
• Need for greater information
• Issue of option of treatment-free death
• Physician should discuss
• Ending treatment does not mean You are a bad
  parent.
• Ending treatment is not giving up.
• No heroic requirement to go down fighting.

11

• To keep the pediatric patient lot improving, the
  cures growing and the effects of therapy on
  quality
• of life, particularly in the hard to handle
  cancers, we need to innovate within the careful,
  patient-centered model that pediatricians have
  always utilized.
• Grace Monaco

12
Barriers to phase 1 pediatric

• Industry often un-enthusiastic
• Rare pediatric-specific tumor types
• Small childhood populations
• Problematic access to clinical trial
  information
• Health insurance/billing concerns
• Centralized trial information

13
Innovations Pediatric Tumors

• Perception/action on phase 1 drugs may evolve
• Disparate adult/child tumor types will show
  similar drug-responsive pathways
• Adult CML vs. pediatric medulloblastoma Gleevec
• Emphasis on cross-correlation of genome anatomies
  (adult, pediatric)
• mRNA expression profiles
• Genomic DNA somatic mutations
• Ensure that pediatric tumors represented in
  Cancer Genome Anatomy Program (CGAP) and other
  genomic initiatives.

14
Adult phase 1 prior to kids?

• There is no reason that the pediatric oncology
  community should wait for results from any adult
  trial before designing their own Phase Is and
  pilots for the use of new and old agents in
  pediatric oncology.
• Grace Monaco

15
Adult phase 1 before kids?

• In some cases?
• Basis for dose initiation
• reduce under-dosing/over-dosing of phase I trials
• Safety testing

16
Small Pediatric Population

• Formalize coordination of US Adult Cooperative
  Group Clinical Trial Studies and
  COG/Academic/Pharmacy Child Studies for
  Simultaneous Access
• Yearly joint symposium on phase 1 trials
• Address Phase 1/in conjunction with palliative
  care Discussions/documents/consents/assents
• Address Emerging targeted pathways shared by
  tumors (sub-committees/consortia, data sharing)

17
Small Pediatric Population

• IF pediatrics is to benefit from greater numbers
  of adult phase 1 trials
• Need much better participation of adults in adult
  trials
• US adult participation in cancer clinical trials
  5
• Canadian adult participation 65
• Canadian adult cancer patients gt90 adults
  treated at Comprehensive Cancer Centers

18
Innovations continued

• I am writing on behalf on my friend's sick
  child. Could you please send me some information
  on international treatment resources available
  for a child who has leukemia, acute lymphoblastic
  form. This is a boy and he lives in Ukraine.
  Resources are limited there, but I heard that in
  Russia some clinics successfully treat this
  disease. If you need more information about him,
  please let me know. Please, help us to find a
  way to help the child.

19
Innovations continued

• Increase collaborative international research
  through international clinical trial
  participation
• (Internationally, only 1 in 5 children have
  access to childhood cancer treatment.)

20
Access to Information

• Utilization of common, comprehensive
  child-specific clinical trial information service
  by academia, COG, individual institutions and
  industry.
• Funding of such clinical trial information
  services (such as HopeLink).

21
"Life isn't measured by the number of breaths we
take, but by the moments that take our breath
away."